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Peking Union Medical College Hospital — Rare Disease Pipeline | Kisho

Peking Union Medical College Hospital

Other

Orphan Designations

0 approved, 1 designated

FDA Approvals

Active Trials

200 recruiting

Rare Diseases

across 29 areas

News (30d)

Quiet

Peking Union Medical College Hospital is a company with 1 orphan drug designation across 18 rare diseases. Active clinical trials in 28 indications. 14 active policy bills affect their portfolio.

Competitive Landscape

Diversified Portfolio

Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.

Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA

Approved: 0

In Trials: 15

Designated Only: 3

18 total diseases

Disease	Drug(s)	Stage
Alzheimer disease	-	Des.TrialAppr.
Duchenne muscular dystrophy	-	Des.TrialAppr.
Leber congenital amaurosis 13	AAV-hRDH12: Recombinant adeno-associated virus vector expressing human Retinol dehydrogenase 12 (RDH12) gene	Des.TrialAppr.
RDH12-related dominant retinopathy	AAV-hRDH12: Recombinant adeno-associated virus vector expressing human Retinol dehydrogenase 12 (RDH12) gene	Des.TrialAppr.
RDH12-related recessive retinopathy	AAV-hRDH12: Recombinant adeno-associated virus vector expressing human Retinol dehydrogenase 12 (RDH12) gene	Des.TrialAppr.
amyloidosis	-	Des.TrialAppr.
brain neoplasm	-	Des.TrialAppr.
breast cancer	-	Des.TrialAppr.
cancer	-	Des.TrialAppr.
cardiovascular disorder	-	Des.TrialAppr.
cervical cancer	-	Des.TrialAppr.
cystic fibrosis	-	Des.TrialAppr.
diabetes mellitus	-	Des.TrialAppr.
lupus erythematosus	-	Des.TrialAppr.
lymphoma	-	Des.TrialAppr.
muscular dystrophy	-	Des.TrialAppr.
ovarian cancer	-	Des.TrialAppr.
sarcoma	-	Des.TrialAppr.